Rare Disease Report

Origins of the United Pompe Foundation

APRIL 20, 2017
David Hamlin

David Hamlin describes the origins of the United Pompe Foundation and how it helps families affected by Pompe Disease.

As David explains in this clip, the foundation was formed to learn more about Pompe disease and meet other people managing this very rare condition. The foundation provides information and guidance for families and patients with Pompe disease.

We talked to David at Duke University's late-onset Pompe disease patient meeting held April 9, 2017. 

About Pompe Disease

Pompe disease is a rare lysosomal disease that may present in childhood (early-onset) or in adulthood (late-onset). In both cases, the disease is due to a deficiency in the enzyme alpha-glucosidase (GAA). Symptoms vary greatly in this population but can be severe and ultimately deadly in the early-onset type.


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